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Targeted Therapy
Source: Childhood Basal Cell Carcinoma and Squamous Cell Carcinoma of the Skin Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: September 11, 2024 · Captured 2026-09-09.
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Context: Treatment of Childhood Basal Cell Carcinoma (BCC) and Squamous Cell Carcinoma (SCC) of the Skin
Most BCCs have activation of the hedgehog pathway, generally resulting from variants in PTCH1. For more information about PTCH1, see Genetics of Skin Cancer.[2]
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Vismodegib. In adults, vismodegib (GDC-0449), a hedgehog pathway inhibitor, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of metastatic or advanced BCC.[3-5] This drug also reduces the tumor burden in patients with basal cell nevus syndrome.[6] In a case report, one child with xeroderma pigmentosum and a nodular BCC achieved a complete clinical response with vismodegib.[7]
Cemiplimab. No prospective trials of cemiplimab (PD-1 inhibitor) have been conducted in children and adolescents. However, the FDA approved cemiplimab for use in patients with metastatic or locally advanced cutaneous SCC who are not candidates for surgery or radiation and in patients with locally advanced or metastatic BCC previously treated with a hedgehog pathway inhibitor or for whom a hedgehog pathway inhibitor is not appropriate.[8,9]
For more information, see Skin Cancer Treatment.
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Preserved source evidence · Not medical advice